Neovascular Age-related Macular Degeneration
Conditions
Keywords
AMD, wet AMD, wAMD, nAMD
Brief summary
This is a phase 1/2 clinical study to evaluate the safety, preliminary efficacy, immunogenicity, and pharmacokinetic (PK) characteristics of SKG0106 in subjects with nAMD. Based on results from the phase 1 dose escalation study, the phase 2 expansion study will be conducted.
Interventions
GENETICSKG0106
SKG0106 is a recombinant adeno-associated virus (AAV) vector-based in vivo gene therapeutic product
Sponsors
Skyline Therapeutics (US) Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Voluntary and able to sign a dated ICF prior to any study-related procedures and able to complete the study as required by the protocol; 2. Aged ≥ 50 years at screening; Study Eye: 3. Diagnosis of nAMD as determined by the PI; 4. Active CNV lesions secondary to age-related macular degeneration (AMD); 5. Subjects must have been responsive to anti-VEGF therapy as assessed by the PI prior to study treatment.
Exclusion criteria
1. Any active intraocular or periocular infection or active intraocular inflammation (e.g., infectious conjunctivitis, keratitis, scleritis, endophthalmitis, infectious blepharitis, uveitis) in the study eye at baseline; 2. Retinal pigment epithelial tear in the study eye at screening; 3. Current vitreous hemorrhage in the study eye or history of vitreous hemorrhage within 4 weeks prior to baseline; 4. Any condition that, in the opinion of the investigator, may limit visual acuity improvement in the study eye; 5. History of retinal detachment or active retinal detachment in the study eye; 6. Any prior gene therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Characteristics of dose limiting toxicities (DLTs) | 4 Weeks |
| Type, severity, and incidence of ocular and systemic adverse events (AEs) | 52 Weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean change from baseline in best corrected visual acuity (BCVA) at each visit | 52 Weeks | BCVA of the study eye is examined by the Early Treatment of Diabetic Retinopathy Study (ETDRS) testing at screening and baseline. |
| Mean change from baseline in central subfield thickness (CST) at each visit | 52 Weeks | Spectral domain optical coherence tomography (SD-OCT) will be performed to measure CST at screening and at each visit (prior to study drug injection, if available). |
| Mean change from baseline in patient-reported outcome (VFQ-25) scale score at each visit | 52 Weeks | Both the total scale and subscale scores of the VFQ-25 is ranging from 0 to 100, and higher scores mean a better outcome. |
Countries
China, United States
Contacts
CONTACTEddy Anglade, MD
Outcome results
None listed